Cambridge, Mass.
Biogen Inc. (Nasdaq: BIIB) today announced that The Journal of the American Medical Association (JAMA) Neurology has published final long-term results from the Phase 3 VALOR study and its open-label extension (OLE) evaluating QALSODY® (tofersen) for the treatment of SOD1-ALS over 3.5 years of follow-up. The results demonstrate that early initiation of QALSODY is associated with slower decline in clinical function, breathing, and strength, as well as a reduced risk of death or permanent ventilation. Sustained reductions in neurofilament, a biomarker of neurodegeneration, further validate the drug’s impact on disease biology.
A subset of QALSODY-treated participants experienced regained muscle strength and function, a phenomenon not previously reported in the natural history of SOD1-ALS.
Key Highlights:
- Early-start QALSODY participants showed measurable improvements in muscle strength over ~3 years.
- Neurofilament reductions confirmed biological impact on SOD1-ALS.
- Safety profile remains consistent with prior studies; most common adverse events were headache, procedural pain, and back or extremity pain. Serious neurological events were rare and manageable.
Study Overview:
- VALOR: Phase 3, six-month, randomized, double-blind, placebo-controlled trial. 108 participants (72 QALSODY, 36 placebo).
- OLE: 95 participants from VALOR enrolled; median follow-up 4.9 years (range 3.6–5.4 years).
Expert Insights:
Timothy Miller, M.D., Ph.D., VALOR principal investigator, emphasized:
“In the past, conversations with people living with SOD1-ALS focused on managing progression. Today, QALSODY offers the potential to maximize improvement.”
Merit Cudkowicz, M.D., co-principal investigator, added:
“Starting QALSODY just 6 months earlier in faster-progressing participants was associated with a 3.4-year extension of event-free survival.”
About QALSODY® (tofersen):
QALSODY is an antisense oligonucleotide designed to reduce toxic SOD1 protein production in adults with ALS caused by SOD1 gene mutations. Administered intrathecally, QALSODY has received approval in 44 countries, including accelerated approval in the U.S. based on reductions in plasma neurofilament light chain (NfL). Biogen licensed tofersen from Ionis Pharmaceuticals, which discovered the therapy.
Ongoing Research:
- ATLAS Study: Phase 3, randomized, placebo-controlled trial evaluating presymptomatic initiation of QALSODY to delay ALS onset in individuals with SOD1 mutations. Primary endpoint: emergence of clinically manifest ALS.
About ALS and SOD1-ALS:
Amyotrophic lateral sclerosis (ALS) is a progressive, fatal neurodegenerative disease causing motor neuron loss. SOD1-ALS accounts for ~2% of ALS cases (~330 people in the U.S.), with over 15% of ALS cases linked to genetic mutations. Average life expectancy is 3–5 years post-symptom onset.
Biogen’s Commitment:
Biogen continues to invest in ALS research, with a robust discovery pipeline targeting genetic and sporadic forms of ALS, including TDP-43 pathology seen in 97% of ALS cases.
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